Our “Alnylam 5×15″ and genetic medicine strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need.
Products arising from this strategy share several critical characteristics including:
- the potential to have a major impact in a high unmet need population;
- a genetically defined, liver-expressed target gene involved in disease and validated in human genetics;
- the ability to leverage the existing Alnylam RNAi delivery platform;
- the opportunity to monitor an early, blood-based biomarker with strong disease correlation in Phase 1 clinical trials for human proof of concept; and,
- the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated paths for commercialization.